Posts by: Alice Melão

News

Heart problems are less common, and milder in severity, in people with Sanfilippo syndrome compared with other forms of mucopolysaccharidosis, a Taiwanese study has found. The cardiac problems identified ... Read more

May 9, 2019May 9, 2019

Tissue Remodeling, Toxic Molecule Accumulation Responsible for Upper Airway Involvement in MPS, Study Finds

News

Active tissue remodeling and treatment-resistant accumulation of toxic molecules are contributing factors in the development of persistent upper airway disease among patients with mucopolysaccharidosis (MPS), a study shows. The ... Read more

April 4, 2019April 4, 2019

Alpha-Synuclein Not Responsible for Early Symptoms of Sanfilippo Type A, Study Shows

News

Alpha-synuclein protein is not responsible for the onset of cognitive and motor symptoms associated with Sanfilippo syndrome type A, a mouse study suggests. The study, “Early disease course is ... Read more

March 22, 2019March 22, 2019

Team Sanfilippo Planning to Launch Phase 2 Trial Testing Trehalose as Sanfilippo Treatment

News

Nonprofit research foundation Team Sanfilippo (TSF) is preparing to launch a Phase 2 clinical trial to explore the safety and efficacy of a common sugar called trehalose as a ... Read more

December 20, 2018December 20, 2018

Abeona Will Include Early-stage MPS IIIA Patients in ABO-102 Gene Therapy Trial

News

Abeona Therapeutics is planning to expand its ongoing ABO-102 gene therapy Phase 1/2 trial to include patients with Sanfilippo syndrome type A (MPS IIIA) at earlier stages of the ... Read more

December 13, 2018December 13, 2018

New Stem Cell Lines Derived From MPSIIIB Patient May Help Improve Preclinical Research

News

Two new experimental stem cell lines derived from a patient with mucopolysaccharydosis IIIB (MPSIIIB), also known as Sanfilippo syndrome type B, were developed by Spanish researchers. These stem cells ... Read more

November 8, 2018November 8, 2018

Shire’s Investigational ERT Shows Limited Benefits for Sanfilippo Type A Children in Phase 2b Trial

News

Administration of an engineered version of the heparan-N-sulfatase (HNS) enzyme into the cerebrospinal fluid directly through the spinal canal failed to improve neurocognitive activity in infants with early-stage Sanfilippo syndrome ... Read more

September 25, 2018September 25, 2018

More Studies Needed to Characterize Features, Course of MPS III, Review Says

News

Very little is still known about the clinical features and natural course of Sanfilippo syndrome, also known as mucopolysaccharidosis (MPS) III, a rare disease that can significantly affect children’s ... Read more

September 13, 2018September 13, 2018

Metabolomics Study Helps Researchers Get Closer to Identifying Sanfilippo Biomarkers

News

A comprehensive analysis of compounds present in the urine of patients with Sanfilippo syndrome provides new insight into the biological processes likely involved in the disease. The research could help ... Read more

June 27, 2018June 27, 2018

Common Sugar, a Food Additive, Seen to Ease Brain Inflammation in Mouse Model of Sanfilippo IIIB

News

A common sugar often used as a food additive was seen to ease inflammation in the brain and retina, and extend the life of Sanfilippo disease IIIB (mucopolysaccharidosis IIIB) mice in ... Read more